BioCentury | Sep 29, 2020
Distillery Therapeutics

Small molecule kinase inhibitor for cystic fibrosis with splicing mutation

DISEASE CATEGORY: Pulmonary INDICATION: Cystic fibrosis (CF) A small molecule inhibitor of four kinases that regulate splicing could treat CF caused by the most common disease-associated CFTR splicing...
BioCentury | Jan 13, 2020
Company News

For $75M up front, Biogen will pursue Pfizer castoff for circadian rhythm in AD, PD

Biogen acquired a clinical asset from Pfizer and will investigate its potential to normalize circadian rhythm to treat “sundowning” in Alzheimer’s disease and irregular sleep associated with Parkinson’s disease. Monday’s deal is Biogen’s second in...
BioCentury | Jan 10, 2020
Product Development

Seeking structural endpoints for OA

As the first disease-modifying therapies for osteoarthritis enter late-stage development, companies are testing new endpoints that can capture effects on joint structure instead of relying on traditional pain-related readouts. Most are converging on MRI imaging...
BioCentury | Aug 23, 2019
Targets & Mechanisms

New targets in osteoarthritis are about more than just pain

New biological insights and increased recognition from FDA are transforming osteoarthritis from a testing ground for new pain therapies into its own high-priority indication. At least two mechanisms with disease-modifying potential for osteoarthritis (OA) have...
BioCentury | Feb 8, 2019
Clinical News

Samumed unveils MOA for Wnt inhibitor OA therapy

Samumed LLC (San Diego, Calif.) reported preclinical data showing that its small molecule Wnt pathway inhibitor osteoarthritis therapy, SM04690, functions through CLK-2 and DYRK1A inhibition. Data were presented at the Orthopaedic Research Society meeting in...
BioCentury | Jul 31, 2017
Distillery Therapeutics

Hepatic

INDICATION: Liver disease In vitro and mouse studies identified a triazoloquinolone-based CLK-1 inhibitor that could help treat acute liver injury (ALI). In silico screening of small compound libraries, chemical synthesis of analogs of the screening...
BioCentury | Jun 9, 2016
Distillery Therapeutics

Therapeutics: Calmodulin; CDC-like kinase 1 (CLK-1); vascular endothelial growth factor receptor 3 (FLT4; VEGFR-3); K(lysine) acetyltransferase 5 (KAT5); acetyl

Infectious disease INDICATION: Chikungunya virus Cell culture and mouse studies identified inhibitors of calmodulin , CLK-1 , FLT4, KAT5 and other host factors that could help treat Chikungunya viral infection. Screening of an siRNA library targeting about...
BioCentury | Mar 3, 2016
Targets & Mechanisms

Rescue mission

A pair of preclinical studies published last month on the autism target SHANK3 provide what might be the most meaningful progress for patients since the gene was linked with the disease over a decade ago....
BioCentury | Feb 25, 2016
Distillery Therapeutics

Therapeutics: Protein kinase B (AKT; AKT1; PKB; PKBA); CDC-like kinase 2 (CLK-2); SH3 and multiple ankyrin repeat domains 3 (SHANK3; PROSAP2; SPANK-2)

Neurology INDICATION: Autism Patient sample and rodent studies suggest inhibiting CLK-2 or activating AKT could help treat Phelan-McDermid syndrome (PMDS) or autism in patients harboring SHANK3 mutations. Levels of T308-phosphorylated AKT were lower in induced...
BioCentury | Sep 17, 2012
Product Development

Excited about cycling

New synthesis and screening technologies and the allure of access to previously undruggable targets are driving an explosion of new company formation and deal-making around macrocycles and constrained peptides. While it remains to be seen...
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